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Paper of the Month: Stem cell therapy for retinal degeneration

07/07/10 Portland, OR

This month's featured paper, published in PLoS One is: "Non-Invasive Stem Cell Therapy in a Rat Model for Retinal Degeneration and Vascular Pathology," by a group of ten investigators from the OHSU Casey Eye Institute and the Oregon Stem Cell Center. The authors are: Shaomei Wang, Bin Lu, Sergei Girman, Jie Duan, Trevor McFarland, Qing-shuo Zhang, Markus Grompe, Grazyna Adamus, Binoy Appukuttan and Raymond Lund. This research highlights the collaborative work of two important research groups at OHSU.

Retinal degeneration is largely untreatable and affects millions of people in the U.S. This research focuses on using adult stem cells to develop a novel treatment for retinal degeneration. Led by Dr. Wang, the study explores the potential of systemic administration of pluripotent bone marrow-derived mesenchymal stem cells (MSCs) to treat retinal degeneration and associated vascular pathology in the Royal College of Surgeons (RCS) rat, a well-established animal model for retinitis pigmentosa. The animals received syngeneic MSCs by tail vein at an age before major photoreceptor loss.

The results showed that both rod and cone photoreceptors were preserved (5 - 6 cell layers) at the time when only a single layer of photoreceptors remained in the control animals, and visual function was significantly preserved compared with the controls. The number of pathological vascular complexes (abnormal vessels associated with migrating pigment epithelium cells) and areas of vascular leakage that would ordinarily develop were dramatically reduced. RT-PCR analysis and immunohistochemistry revealed that there was upregulation of growth factors and neurotrophic factors in the eyes of animals that received MSCs.

Pluripotent MSCs have been widely used in both autologous and allogeneic settings for clinical trials for degenerative and regenerative medicine because they are relatively easy to isolate, they secrete large quantities of bioactive factors that are both immunomodulatory and trophic, and they raise fewer ethical concerns than other stem cell choices. The fact that MSCs can be administered systemically is a huge advantage, as repeat injections could be performed without damaging the eye, making long-term treatment possible.

Dr. Wang has recently submitted an RO1 application for NIH support to continue this line of research. If positive results are obtained in animal models, this treatment has a realistic likelihood of translation to clinical applications.

The School of Medicine News spotlights a recently published faculty research paper in each issue. The goals are to highlight the great research happening at OHSU and to share this information across departments, institutes and disciplines. The paper is selected by Associate Dean for Basic Science Mary Stenzel-Poore. This monthly paper summary is reviewed prior to publication by Dean Mark Richardson and Vice President/Senior Associate Dean for Research Dan Dorsa.

A list of all papers published by OHSU authors during the prior month as compiled by the OHSU Library is provided here.